Study met its primary and key secondary endpoints, demonstrating rapid and sustained improvement in near visual acuitySafety profile consistent with previous clinical trials and no treatment-related serious adverse events reported in the studyNo evidence of tachyphylaxis was observed in this study over the 6-week periodManagement to Host Webcast and Conference Call Today at 8:00 A.M. ET RESEARCH TRIANGLE PARK, N.C., June 26, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage biopha ...

Core Insights - Edgewise Therapeutics has reported positive results from its sevasemten program for Becker and Duchenne muscular dystrophies, demonstrating sustained disease stabilization and a clear path to potential registration as the first therapy for Becker [1][3]. Group 1: Becker Muscular Dystrophy - The MESA trial data showed that 99% of eligible participants (n=85) are enrolled, with significant improvements in North Star Ambulatory Assessment (NSAA) scores, indicating sustained disease stabilization over 18 months [2]. - Participants from the CANYON trial who transitioned to MESA exhibited an average NSAA score improvement of 0.8 points from baseline, while those on placebo showed a 0.2 point improvement after starting sevasemten [2]. - The FDA has provided a clear path for sevasemten registration, emphasizing the importance of NSAA as a clinically meaningful endpoint for traditional approval [3]. Group 2: Duchenne Muscular Dystrophy - Encouraging topline data from Phase 2 trials (LYNX and FOX) indicate that sevasemten is well-tolerated and shows potential for reducing functional decline at a target dose of 10 mg [4][6]. - The LYNX trial focuses on four- to nine-year-old participants, while the FOX trial involves six- to fourteen-year-old participants previously treated with gene therapy, both assessing safety and biomarkers of muscle damage [5][6]. - The company plans to meet with the FDA in Q4 2025 to discuss Phase 3 design, aiming to initiate the pivotal study in 2026 [7]. Group 3: Company Overview - Edgewise Therapeutics is a biopharmaceutical company focused on developing novel therapeutics for muscular dystrophies and cardiac conditions, with sevasemten being a first-in-class fast skeletal myosin inhibitor [9][12]. - The company has achieved significant regulatory milestones, including FDA Orphan Drug Designation and Fast Track designations for both Becker and Duchenne [9]. - Edgewise is dedicated to changing the lives of patients affected by serious muscle diseases, with a strong emphasis on innovative therapeutic solutions [12].

Core Viewpoint - Altimmune, Inc. is set to present topline data from its IMPACT Phase 2b trial evaluating pemvidutide for treating metabolic dysfunction-associated steatohepatitis (MASH) on June 26, 2025 [1][2]. Group 1: Company Overview - Altimmune is a late clinical-stage biopharmaceutical company focused on developing novel peptide-based therapeutics for liver and cardiometabolic diseases [5]. - The company's lead program, pemvidutide, is a GLP-1/glucagon dual receptor agonist aimed at treating MASH, obesity, Alcohol Use Disorder (AUD), and Alcohol-associated Liver Disease (ALD) [5]. Group 2: IMPACT Study Details - The IMPACT trial enrolled 212 patients with biopsy-confirmed MASH and fibrosis stages F2/F3, randomized to receive either weekly subcutaneous pemvidutide at doses of 1.2 mg and 1.8 mg or placebo for 24 weeks [3]. - Key efficacy endpoints include MASH resolution or fibrosis improvement at 24 weeks, with secondary endpoints focusing on weight loss and non-invasive tests [3]. - A total of 48 weeks of treatment is planned, with final readout expected in Q4 2025 [3]. Group 3: Pemvidutide Characteristics - Pemvidutide is an investigational peptide-based dual receptor agonist that mimics the effects of diet and exercise on weight loss, suppressing appetite and increasing energy expenditure [4]. - Clinical trials have shown significant MASH resolution, liver fibrosis improvement, and weight loss, along with reductions in liver fat content, triglycerides, LDL cholesterol, and blood pressure [4]. - The U.S. FDA has granted Fast Track designation to pemvidutide for MASH treatment, with ongoing trials for AUD and ALD [4].

Core Viewpoint - Appili Therapeutics has successfully re-launched LIKMEZ™ (ATI-1501), a metronidazole oral suspension, and is actively pursuing funding opportunities to support its development programs and enhance its market position [2][3][14]. Group 1: Product Development and Market Position - LIKMEZ™ (ATI-1501) is the first FDA-approved, ready-to-use liquid oral suspension of metronidazole, specifically designed for patients who have difficulty swallowing tablets or are sensitive to taste [3][4]. - The re-launch of LIKMEZ follows new patent coverage extending to 2039, marking a significant milestone for the company and its partner, Saptalis Pharmaceuticals [2]. - Appili is focused on driving continued sales of LIKMEZ while advancing other product candidates, including ATI-1701 and ATI-1801 [3]. Group 2: Funding and Financial Performance - The company has submitted five non-dilutive funding proposals to the U.S. government, with a total potential value of up to US$125 million, aimed at supporting the development of critical infectious disease products [3][14]. - For the fiscal year ended March 31, 2025, Appili reported a net loss of CAD 2.6 million, which is an improvement from a net loss of CAD 3.8 million in the previous year, primarily due to increased government assistance and other income [19][20]. - As of March 31, 2025, the company had cash reserves of CAD 1.2 million, a significant increase from CAD 0.1 million a year earlier, indicating improved liquidity [21]. Group 3: Research and Development Progress - ATI-1701, a biodefense vaccine candidate for tularemia, has received approximately US$11.6 million in funding from the U.S. Air Force Academy and has shown promising results in providing protection against lethal tularemia [5][6]. - The company is actively pursuing additional funding and partnership opportunities for ATI-1801, a topical antiparasitic product that has received Orphan Drug Designation from the FDA [10][13]. - Appili has successfully completed technology transfer for the ATI-1701 manufacturing process, supporting future development activities [8].

Core Insights - Vor Bio has secured exclusive global rights (excluding Greater China) to develop and commercialize telitacicept, a dual-target recombinant fusion protein for autoimmune diseases [1][6] - RemeGen has received an initial payment of $125 million, which includes a $45 million upfront payment and $80 million in warrants, along with potential milestones exceeding $4 billion and tiered royalties [1][5] - Jean-Paul Kress, MD, has been appointed as the new CEO and Chairman of Vor Bio, bringing extensive experience in clinical development and commercialization [3][4] Company Developments - Vor Bio is focused on advancing telitacicept through Phase 3 clinical development to address serious autoantibody-driven conditions globally [7] - RemeGen is conducting a global Phase 3 clinical trial for telitacicept, with initial results expected in the first half of 2027 [2][6] - The strategic out-licensing of telitacicept's ex-China rights is aimed at maximizing its clinical and commercial potential on a global scale [5] Product Information - Telitacicept targets key immune pathways by inhibiting BlyS (BAFF) and APRIL, which are critical for B cell survival, thereby reducing autoreactive B cells and autoantibody production [2][5] - In a Phase 3 trial in China for generalized myasthenia gravis, telitacicept showed a 4.8-point improvement in the MG-ADL scale compared to placebo at 24 weeks [5]

Core Insights - Kodiak Sciences Inc. is presenting KSI-101, a bispecific investigational biologic targeting macular edema secondary to inflammation, at the 2025 Congress of the International Ocular Inflammation Society in Rio de Janeiro [1][6] - The company aims to address the significant unmet need in treating macular edema caused by inflammation, which can lead to vision loss [3][4] Company Overview - Kodiak Sciences is a precommercial biotechnology company focused on developing therapeutics for retinal diseases, utilizing its ABC Platform for innovative drug design [8][9] - The company is advancing its pipeline, which includes three late-phase clinical assets expected to deliver Phase 3 topline data in 2026 [9] Product Details - KSI-101 is a high-strength (100 mg/mL) bispecific protein designed to target both interleukin-6 (IL-6) and vascular endothelial growth factor (VEGF), addressing the dual mechanisms of macular edema [6][5] - The ongoing Phase 1b study, APEX, is evaluating the safety and tolerability of KSI-101, with plans to progress into dual Phase 2b/3 studies [7]

Core Viewpoint - Reviva Pharmaceuticals Holdings, Inc. plans to offer shares of its common stock and warrants in a public offering to fund research and development activities and for general corporate purposes [1][2]. Company Overview - Reviva is a late-stage biopharmaceutical company focused on developing therapies for unmet medical needs in central nervous system (CNS), inflammatory, and cardiometabolic diseases [5]. - The company's pipeline includes two drug candidates, brilaroxazine (RP5063) and RP1208, both of which are new chemical entities discovered in-house and have been granted composition of matter patents in multiple regions [5]. Offering Details - The offering will be conducted under an effective shelf registration statement on Form S-3, filed with the U.S. Securities and Exchange Commission (SEC) [3]. - A.G.P./Alliance Global Partners is acting as the sole placement agent for the offering [2].

Châtillon, France, June 25, 2025 DBV Technologies Announces First Subject Screened in COMFORT Toddlers Supplemental Safety Study in Peanut Allergic Toddlers 1 – 3 Years Old First subject screened at the Respiratory Medicine Research Institute of Michigan with Dr. Jeffrey Leflein acting as Principal InvestigatorAdditional sites, including Allergy and Asthma Center of Minnesota and Hamilton Allergy and Immunology Clinic of Ontario, Canada have been activated and are scheduling screenings DBV Technologies (Eu ...

Châtillon, France, June 25, 2025 DBV Technologies Announces First Subject Screened in COMFORT Toddlers Supplemental Safety Study in Peanut Allergic Toddlers 1 – 3 Years Old First subject screened at the Respiratory Medicine Research Institute of Michigan with Dr. Jeffrey Leflein acting as Principal InvestigatorAdditional sites, including Allergy and Asthma Center of Minnesota and Hamilton Allergy and Immunology Clinic of Ontario, Canada have been activated and are scheduling screenings DBV Technologies (Eu ...

Core Viewpoint - Idorsia Ltd. successfully held bondholder meetings to approve amendments to the terms of its convertible bonds, extending their maturity dates significantly [1][2][3]. Group 1: Bondholder Meetings - Two bondholder meetings were conducted for Idorsia's CHF 200 million convertible bonds maturing in 2025 and CHF 600 million convertible bonds maturing in 2028 [2]. - The amendments include extending the maturity date of the CB 2025 to July 17, 2034, and the CB 2028 to August 4, 2038 [2]. Group 2: Voting Outcomes - A significant majority of 89.5% of CB 2025 holders and 93.5% of CB 2028 holders voted in favor of the amendments, surpassing the required two-thirds majority [3]. - The amendments will become binding upon approval by the higher cantonal composition authority and are contingent on the completion of the repurchase offer for both bonds [3]. Group 3: Company Overview - Idorsia Ltd. aims to challenge medical paradigms and develop transformative medicines, positioning itself as a leading biopharmaceutical company [6]. - The company is headquartered near Basel, Switzerland, and has a strong focus on small-molecule drug discovery [7].